Avrion Therapeutics targets genetically-linked neurodegenerative diseases using precision gene therapy to enable long-term solutions.
Founded in 2020, based on more than 10 years of research at the Swiss Federal Institute of Technology EPFL Brain Mind Institute, Avrion says its first-in-class precision gene therapy platform can selectively and safely target discrete cells in the brain including neurons and astrocytes.
The gene therapy technology utilises vectors to deliver therapeutic gene sequences to discrete cell types in the central nervous system. Avrion’s first clinical candidate gene therapy product, AVR-001, is preparing to enter IND-enabling studies to treat patients with a form of motor neuron disease (amyotrophic lateral sclerosis – ALS or MND), a fatal neuromuscular disorder.
The company says that there are currently just two drugs on the market which bring only marginal benefits to ALS patients, and no cure. Avrion hopes its precision gene therapy approach will bring transformational solutions to people with ALS.
The therapeutic platform is supported by a preclinical discovery engine to identify novel targets to treat neurodegenerative diseases with unmet medical need. The company says these targets will be addressed by its technology which allows cell-specific genetic manipulation within the brain and spinal cord.
The company notes that many of the hurdles faced over recent decades with gene therapies have been circumvented with the introduction of Adeno-Associated Virus (AAV) vectors. Avrion’s co-founder and Chief Development Officer, Dr. Harald Petry, is the former CSO at uniQure and has previously brought an AAV-based gene therapy product to the market (Glybera).
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